Local researchers working to cure cancer

Pre-clinical trial experiments show positive signs of tumor reduction

When one thinks “chemo,” mental images of hair loss, nausea or cachexia, also known as wasting away syndrome, are easy to conjure.

But local researchers might have a less severe solution.

Through some pre-clinical trial experiments, fenretinide and safingol showed positive signs of cancerous tumor reduction, said Bill Simpson, chief operating officer of CerRx, the company working with the drugs.

Drs. Patrick Reynolds and Barry Maurer, Texas Tech Health Sciences Center cancer researchers, established CerRx in 2008 as a developmental research company for anticancer therapeutics focused on ceramides — wax-like building blocks in cells that signal cell production operations, according to a news release from the company. The CerRx products work by essentially tricking cancerous cells into self-destruction.

The two products CerRx officials are focused on include fenretinide and safingol, Simpson said.

“For lots of pre-clinical and scientific rationale animal models, they were convinced that fenretinide and safingol were likely to contribute significantly to the treatment of cancer,” Simpson said.

The project goal is to combine fenretinide and safingol and administer it by IV to patients to manipulate cancer cells into self-destruction without harming healthy cells, he said.

“Cancer cells are really smart,” Simpson said. “That’s why a lot of people benefit and then they fail. So the cancer cell figures out, ‘This drug is no good for me.’ So you put the drug in the body, the cancer cell absorbs it, the tumor cells die and the tumor cells figure out that the substance is no good and says, ‘I’m not letting you into my cells.’ That’s making it really simplistic. It’s really complicated.”

The fenretinide portion of the therapeutic chemo allows treatment to focus on just cancer cells, Simpson said.

“The study will be used to combine fenretinide, that we believe and we’ve actually seen has quite a bit of activity in shrinking and making tumors disappear, we intend to combine that with our second compound — safingol,” Simpson said.

“… We believe that the combination of these two agents will give us even better results than we have seen.”

The safingol portion of the compound works to manipulate the cell into keeping the cancer-fighting drug within the cell, Simpson said.

Earlier this month, CerRx received a $6 million grant from the Cancer Prevention and Research Institute of Texas. That money, in addition to grants from angel investors, will advance the trial of the drug compound. It’s in phase one of drug development, he said.

With the phase one part of the study, Simpson said the goal is to figure out the right dose.

“You usually start at a low dose and increase the dose in a logical progression until you start getting a lot of toxicity,” he said. “From there, you figure out what is the safe dose to give, but is still a high enough dose that it’s likely to give you the efficacy or the response of the shrinkage of the tumor that you’re looking for.”

Simpson said CerRx officials have been surprised by the response in the first phase of the study. Patients don’t usually yield much response within this phase, he said.

In a provided statement, Reynolds, chief scientific officer of CerRx, said: “IV fenretinide has demonstrated substantial activity in T-cell lymphoma, and phase one results in solid tumors are also encouraging with signals of activity in adenocarcinomas of the esophagus and colon, and in children with neuroblastoma.”

Simpson said side effects of the compound are different than side effects associated with traditional chemotherapy treatments.

“I grew up with traditional chemotherapies,” Simpson said. “Drugs like Adriamycin, one that’s still used, very active, but very, very potent and causes a lot of toxicity. Fenretinide, because of its unique mechanism, seems to only target cancer cells and leaves the normal cells alone. So unlike the traditional chemotherapies, patients don’t lose their hair.”

Patients are also less likely to develop infections, he said.

“They don’t get hospitalized as often, they don’t seem to get nausea and vomiting or diarrhea,” Simpson said.

The drug has been tested on few patients so far, he said. The drug is not flawless. Two of the more common and expected side effects include high cholesterol and low night visibility.

Officials are satisfied with what they’ve seen so far, and they remain optimistic about the fenretinide compound’s future, he said. The drug has been studied on leukemia and lymphoma patients so far, he said. In each study, patients showed partial or complete remission after use of the compound.

“We believe we have a winner on two fronts — one from the activities side and one from the side effect profile,” he said.